RARE Daily

Gilead Granted Breakthrough Therapy Designation for Treatment for Myelodysplastic Syndrome

September 15, 2020

Rare Daily Staff

The U.S. Food and Drug Administration granted Breakthrough Therapy designation for Gilead Sciences’ magrolimab for the treatment of newly diagnosed myelodysplastic syndrome.

Myelodysplastic syndromes (MDS) are a rare group of blood cancers characterized by abnormal development of blood cells within the bone marrow. Approximately 15,000 people are diagnosed with MDS in the United States each year, and no new treatments have been approved in 14 years. The average survival rate for those with lower-risk MDS is six years and approximately 18 months for those with higher-risk MDS.

Individuals with MDS have abnormally low blood cell levels with symptoms including dizziness, fatigue, weakness, shortness of breath, bruising and bleeding, frequent infections, and headaches. In some cases, MDS may progress to bone marrow failure or an acute leukemia. The exact cause of MDS is unknown. It sometimes runs in families, but no disease-causing gene has been identified. Treatment depends on the affected individual’s age, general health, and type of MDS and may include red cell and/or platelet transfusions and antibiotics.

Breakthrough Therapy designation is designed to expedite the development and regulatory review of experimental treatments for serious or life-threatening conditions that, based on preliminary clinical evidence, have the potential to substantially improve clinical outcomes compared with available therapy.

The FDA granted Breakthrough Therapy designation for magrolimab, a first-in-class experimental anti-CD47 monoclonal and macrophage checkpoint inhibitor, based on positive results of an ongoing phase 1b study, which evaluated magrolimab in combination with azacitidine in previously untreated intermediate, high and very high-risk MDS. In data presented at the 2020 European Hematology Society Congress, 91 percent of evaluable patients (n=33) treated with magrolimab plus azacitidine achieved an objective response, with 42 percent achieving a complete remission. The combination of magrolimab plus azacitidine was generally well-tolerated. No maximum tolerated dose was reached and no MDS patients discontinued treatment due to a treatment-related adverse event.

“The Breakthrough Therapy designation recognizes the potential for magrolimab to help address a significant unmet medical need for people with MDS and underscores the transformative potential of Gilead’s immuno-oncology therapies in development,” said Merdad Parsey, chief medical officer of Gilead Sciences.

Magrolimab is currently being studied in the double-blind, placebo-controlled, randomized phase 3 ENHANCE trial in previously untreated higher risk MDS. The trial will evaluate the safety and efficacy of magrolimab, in combination with azacitidine, as measured by complete response and duration of complete response.

Magrolimab has been granted Fast Track designation by the FDA for the treatment of MDS, acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma. Magrolimab has also been granted Orphan Drug designation by the FDA for MDS and AML and by the European Medicines Agency for AML.

Photo: Merdad Parsey, chief medical officer of Gilead Sciences

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